Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids
Tyler J. Brittain, Seongmin Jang, Gerard Michael Coughlin, Jonathan D. Hoang, Bre’Anna H. Barcelona, Izabela Giriat, Fiona Ristic, Nathan Appling, Camille PMA Chossis, Timothy F. Shay, Viviana Gradinaru
The paper solves cryo-EM structures of AAV capsids and their complexes with AAVR to design variants with reduced liver targeting and altered brain cell tropism.
Molecular Therapy · Mar 28, 2026Peer-reviewedMolecular Biology